Odevixibat for PFIC in China: Real-world Safety and Effectiveness
Official title: Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay).
Run by Ipsen · NCT07588880 · Data updated 2026-11-12
This registry collects real-world safety and effectiveness data on children with PFIC who are being treated with odevixibat in China.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 6 Months and up |
| Sex | All |
| Enrollment | 20 (estimated) |
| Sponsor | Ipsen Industry-sponsored |
| Locations | 6 sites (Beijing and 5 other locations) |
| Status | Recruiting |
| Started | April 16, 2026 |
| Main results expected | April 30, 2031 |
| Study ends | April 30, 2031 |
| Registry record updated | October 23, 2026 |
| Trial ID | NCT07588880 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Children with PFIC of any type who have been prescribed odevixibat by their treating physician and are starting or continuing treatment in China.
✕Probably not a fit
Patients not receiving odevixibat or those not contributing data to the registry may not experience documented benefits.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This registry-based study observes PFIC patients who are prescribed odevixibat as part of routine care in China to understand long-term safety and how well the treatment works in everyday clinical settings. Data will come from participating hospitals to describe adverse events and real-world outcomes over time. The study does not assign treatment or randomize patients, and it includes those starting or continuing odevixibat with consent. It aims to provide a practical picture of how the medicine performs outside of controlled trials.
Why it matters
Potential benefit: If successful, the registry could clarify long-term safety and real-world effectiveness of odevixibat for PFIC in Chinese patients.
How similar studies have gone: Previous registries and real-world studies in PFIC and bile acid transporter therapies have shown that real-world data can reveal safety signals and practical effectiveness, though this specific China PFIC registry is relatively novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enroll the participant in this registry) by their treating physician * On (or starting) active odevixibat treatment (participants can remain in the registry during odevixibat treatment interruptions) * Signed informed consent and assent, as appropriate Exclusion Criteria: * Currently participating in a clinical trial with odevixibat * Currently participating in any interventional clinical trial for PFIC * Have any contraindication to odevixibat as per the approved label in China
Where it's running
-
Hunan Children's Hospital
Hunan, China Recruiting -
Children's Hospital of Fudan University Endocrinology and Metabolism
Shanghai, China Recruiting -
Capital Children's Medical Center
Beijing, China Active not recruiting -
Chongqing Medical University - Children's Hospital
Chongqing, China Active not recruiting -
Nanjing Medical University-Nanjing Children's Hospital
Nanjing, China Active not recruiting -
Xiamen Children's Hospital
Xiamen, China Active not recruiting
Study contacts
- Study coordinator: Ipsen Clinical Study Enquiries
- Email: Clinical.trials@ipsen.com
- Phone: See e-mail
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.